ATE490321T1 - INDUCIBLE SIRNA EXPRESSION CONSTRUCTS FOR TARGETED GENE TURNING OFF - Google Patents

INDUCIBLE SIRNA EXPRESSION CONSTRUCTS FOR TARGETED GENE TURNING OFF

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Publication number
ATE490321T1
ATE490321T1 AT04702315T AT04702315T ATE490321T1 AT E490321 T1 ATE490321 T1 AT E490321T1 AT 04702315 T AT04702315 T AT 04702315T AT 04702315 T AT04702315 T AT 04702315T AT E490321 T1 ATE490321 T1 AT E490321T1
Authority
AT
Austria
Prior art keywords
expression constructs
targeted gene
sirna expression
inducible sirna
turning
Prior art date
Application number
AT04702315T
Other languages
German (de)
Inventor
Thomas Tuschl
Tilmann Achsel
Reinhard Luehrmann
Jutta Dammann
Original Assignee
Max Planck Gesellschaft
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Max Planck Gesellschaft filed Critical Max Planck Gesellschaft
Application granted granted Critical
Publication of ATE490321T1 publication Critical patent/ATE490321T1/en

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Classifications

    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P29/00—Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agents; Non-steroidal antiinflammatory drugs [NSAID]
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00—Drugs for immunological or allergic disorders
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00—Genetically modified animals
    • A01K2217/05—Animals comprising random inserted nucleic acids (transgenic)
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00—Genetically modified animals
    • A01K2217/07—Animals genetically altered by homologous recombination
    • A01K2217/075—Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • C12N2310/111—Antisense spanning the whole gene, or a large part of it
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering N.A.
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2330/00—Production
    • C12N2330/30—Production chemically synthesised

Abstract

The invention relates to vectors for the inducible expression of RNA molecules in eukaryotic, particularly mammalian cells and transgenic animals.
AT04702315T 2003-01-17 2004-01-15 INDUCIBLE SIRNA EXPRESSION CONSTRUCTS FOR TARGETED GENE TURNING OFF ATE490321T1 (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
EP03001058 2003-01-17
PCT/EP2004/000265 WO2004065613A2 (en) 2003-01-17 2004-01-15 Inducible small interfering rna (sirna) expression constructs for targeted gene silencing

Publications (1)

Publication Number Publication Date
ATE490321T1 true ATE490321T1 (en) 2010-12-15

Family

ID=32748770

Family Applications (1)

Application Number Title Priority Date Filing Date
AT04702315T ATE490321T1 (en) 2003-01-17 2004-01-15 INDUCIBLE SIRNA EXPRESSION CONSTRUCTS FOR TARGETED GENE TURNING OFF

Country Status (6)

Country Link
US (2) US8198077B2 (en)
EP (3) EP2314687B1 (en)
AT (1) ATE490321T1 (en)
DE (1) DE602004030315D1 (en)
ES (1) ES2356910T3 (en)
WO (1) WO2004065613A2 (en)

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ES2336887T5 (en) 2000-03-30 2019-03-06 Whitehead Inst Biomedical Res Mediators of RNA interference specific to RNA sequences
DK2813582T3 (en) 2000-12-01 2017-07-31 Max-Planck-Gesellschaft Zur Förderung Der Wss E V Small RNA molecules that mediate RNA interference
PT1504126E (en) 2002-05-03 2014-06-02 Univ Duke A method of regulating gene expression
WO2004014933A1 (en) 2002-08-07 2004-02-19 University Of Massachusetts Compositions for rna interference and methods of use thereof
US7892793B2 (en) * 2002-11-04 2011-02-22 University Of Massachusetts Allele-specific RNA interference
WO2004047764A2 (en) * 2002-11-22 2004-06-10 University Of Massachusetts Modulation of hiv replication by rna interference
EP2314692B1 (en) * 2003-06-02 2021-02-24 University of Massachusetts Methods and compositions for enhancing the efficacy and specificity of RNAi
US7750144B2 (en) * 2003-06-02 2010-07-06 University Of Massachusetts Methods and compositions for enhancing the efficacy and specificity of RNA silencing
PT1633767T (en) * 2003-06-02 2019-02-27 Univ Massachusetts Methods and compositions for controlling efficacy of rna silencing
US8680063B2 (en) * 2003-09-12 2014-03-25 University Of Massachusetts RNA interference for the treatment of gain-of-function disorders
ES2485848T3 (en) * 2003-09-12 2014-08-14 University Of Massachusetts RNA interference for the treatment of disorders related to function gain
GB0327585D0 (en) * 2003-11-27 2003-12-31 Genomica Sau Inducible nucleic acid expression system
US20060134787A1 (en) 2004-12-22 2006-06-22 University Of Massachusetts Methods and compositions for enhancing the efficacy and specificity of single and double blunt-ended siRNA
US20060069050A1 (en) * 2004-02-17 2006-03-30 University Of Massachusetts Methods and compositions for mediating gene silencing
WO2005079532A2 (en) * 2004-02-17 2005-09-01 University Of Massachusetts Methods and compositions for enhancing risc activity in vitro and in vivo
US20060031945A1 (en) * 2004-08-06 2006-02-09 Yu Shen Inducible expression systems for modulating the expression of target genes in eukaryotic cells and non-human animals
EP2098593B1 (en) * 2005-04-15 2011-12-28 National University Corporation Tottori University hTERT expression regulatory gene
WO2007022506A2 (en) * 2005-08-18 2007-02-22 University Of Massachusetts Methods and compositions for treating neurological disease
EP2386564B1 (en) * 2005-10-01 2014-07-16 Charles Stout Regulatable fusion promoters
JP2010507387A (en) 2006-10-25 2010-03-11 クアーク・ファーマスーティカルス、インコーポレイテッド Novel siRNA and method of using the same
WO2008143774A2 (en) * 2007-05-01 2008-11-27 University Of Massachusetts Methods and compositions for locating snp heterozygosity for allele specific diagnosis and therapy
WO2009026574A2 (en) * 2007-08-23 2009-02-26 Keren Pharmaceutical, Inc. Immunogenic compositions and uses thereof
WO2009039173A2 (en) 2007-09-19 2009-03-26 Applied Biosystems Inc. SiRNA SEQUENCE-INDEPENDENT MODIFICATION FORMATS FOR REDUCING OFF-TARGET PHENOTYPIC EFFECTS IN RNAi, AND STABILIZED FORMS THEREOF
WO2009055724A2 (en) * 2007-10-26 2009-04-30 Cold Spring Harbor Laboratory High throughput methods for functionally determining rna interference efficiency
US8217161B2 (en) * 2008-04-22 2012-07-10 Clemson University Research Foundation Methods of inhibiting multiple cytochrome P450 genes with siRNA
WO2010021718A1 (en) 2008-08-19 2010-02-25 Nektar Therapeutics Complexes of small-interfering nucleic acids
CN102803496A (en) 2009-06-10 2012-11-28 淡马锡生命科学研究院有限公司 Virus induced gene silencing (VIGS) for functional analysis of genes in cotton
WO2011035065A1 (en) 2009-09-17 2011-03-24 Nektar Therapeutics Monoconjugated chitosans as delivery agents for small interfering nucleic acids
US20140090102A1 (en) 2011-01-24 2014-03-27 Stéphane Pien Use of the rd29 promoter or fragments thereof for stress-inducible expression of transgenes in cotton
AU2012272908A1 (en) 2011-06-21 2013-12-19 Alnylam Pharmaceuticals, Inc. Assays and methods for determining activity of a therapeutic agent in a subject
BR112014002988A2 (en) 2011-08-12 2017-03-01 Bayer Cropscience Nv specific expression of transgene protection cell in cotton
AU2013201323A1 (en) 2011-10-12 2013-05-02 Bayer Cropscience Ag Plants with decreased activity of a starch dephosphorylating enzyme
BR112014008895A2 (en) 2011-10-12 2019-09-24 Bayer Cropscience Ag plants with decreased activity of a starch dephosphorylation enzyme
US10202643B2 (en) 2011-10-31 2019-02-12 University Of Utah Research Foundation Genetic alterations in glioma
CA2899928A1 (en) * 2013-02-05 2014-08-14 University Of Georgia Research Foundation, Inc. Cell lines for virus production and methods of use

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US5891718A (en) * 1996-03-27 1999-04-06 Vical Incorporated Tetracycline inducible/repressible systems
US6506559B1 (en) * 1997-12-23 2003-01-14 Carnegie Institute Of Washington Genetic inhibition by double-stranded RNA
US20050148530A1 (en) * 2002-02-20 2005-07-07 Sirna Therapeutics, Inc. RNA interference mediated inhibition of vascular endothelial growth factor and vascular endothelial growth factor receptor gene expression using short interfering nucleic acid (siNA)
IL159015A0 (en) * 2001-05-25 2004-05-12 Genset Sa Polypeptides, their preparation and use
US7195916B2 (en) * 2001-09-13 2007-03-27 California Institute Of Technology Method for expression of small antiviral RNA molecules within a cell
EP1458879A2 (en) * 2001-12-21 2004-09-22 Oxford Biomedica (UK) Limited Method for producing a transgenic organism using a lentiviral expression vector such as eiav
EP1551971B1 (en) * 2002-10-17 2008-12-24 TaconicArtemis GmbH Sirna mediated gene silencing in transgenic animals
US20050071893A1 (en) * 2002-10-17 2005-03-31 Jost Seibler SiRNA mediated gene silencing in transgenic animals
WO2004056964A2 (en) 2002-12-18 2004-07-08 Genpath Pharmaceuticals, Incorporated Vectors for inducible rna interference

Also Published As

Publication number Publication date
EP2333063A1 (en) 2011-06-15
WO2004065613A3 (en) 2004-10-07
EP1583832B1 (en) 2010-12-01
EP2314687A1 (en) 2011-04-27
EP1583832A2 (en) 2005-10-12
US20060212950A1 (en) 2006-09-21
WO2004065613A2 (en) 2004-08-05
EP2314687B1 (en) 2017-12-27
DE602004030315D1 (en) 2011-01-13
US20120255045A1 (en) 2012-10-04
US8198077B2 (en) 2012-06-12
ES2356910T3 (en) 2011-04-14
US8470797B2 (en) 2013-06-25

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